CHOP Researchers Propose New Model for Treating Pediatric Thyroid Cancer

Researchers suggest oncogene-specific targeted therapy before or instead of surgery could lead to better outcomes for those with advanced, invasive disease.

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CHOP Main Hospital Building In a recent review published in JAMA Otolaryngology, Ken Kazahaya, MD, associate director of Pediatric Otolaryngology and co-lead surgeon for the Thyroid Center at Children’s Hospital of Philadelphia (CHOP), along with co-researchers from several collaborating pediatric centers, suggest that pediatric patients with advanced invasive thyroid cancer might benefit from being treated with targeted therapies that induce tumor regression, either before or in lieu of surgery. The researchers say this model of patient treatment could lead to better outcomes for those whose tumors have a high risk of surgical complications.

Most pediatric patients with thyroid cancer are successfully treated with surgery. However, some children have extensive disease, in which the cancerous tumor invades the trachea or esophagus or encases vascular or neural structures. For those patients, surgery is highly complex, and there is an elevated risk of complications.

Early data suggest oncogene-specific targeted therapies are effective at shrinking cancerous tumors in children and adults with minimal adverse effects, but in pediatrics, there is no consensus on which patients would benefit from this treatment. The researchers propose a new model for managing complex thyroid cancer and suggest there is an opportunity to enroll children with advanced invasive disease in clinical trials that explore targeted oncogene therapy either before or instead of surgery.

“The development of new, targeted therapies that are able to reduce tumor size with fewer adverse effects and reactions may provide opportunities to treat pediatric patients who have progressive disease that hasn’t responded to traditional therapy,” said senior author Andrew J. Bauer, MD, Medical Director of the Pediatric Thyroid Center at CHOP. “These novel oncogene-specific targeted therapies also provide an opportunity for a paradigm shift in the treatment of patients who initially present with widely invasive disease at diagnosis, in whom surgery involves higher than usual risk.”

Read more about the study here.

Contact: Kaila Revello, The Children’s Hospital of Philadelphia, 267-426-6054 or